On September 12, 2023 Agilent Technologies, Inc. (NYSE: A) and Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported they have entered into a definitive agreement for the sale of Resolution Bioscience to Exact Sciences (Press release, Agilent, SEP 12, 2023, View Source [SID1234635126]). Financial terms of the agreement were not disclosed and are not material to either party.

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"We are pleased that Exact Sciences is acquiring Resolution Bioscience," said Sam Raha, senior vice president, Agilent, and president, Diagnostics and Genomics Group. "Exact is a leading centralized laboratory for advanced cancer diagnostic testing with significant capabilities and resources in this area, which makes Resolution Bioscience a strategic fit for them. This agreement will enable the talented Resolution Bioscience team to continue their work advancing diagnostic solutions for their customers and patients, a very positive outcome of this transaction."

"We are excited to work with the Resolution Bioscience team to integrate their innovative diagnostics into our Precision Oncology portfolio," said Brian Baranick, general manager of Precision Oncology at Exact Sciences. "Resolution Bioscience’s high-quality liquid therapy selection platform perfectly complements our OncoExTra test, allowing Exact Sciences to help more cancer patients determine their best treatment options."

Resolution Bioscience develops and commercializes next-generation sequencing-based precision oncology solutions. Agilent acquired the company in 2021.

On September 12, 2023 Innocare reported that American Journal of Hematology recently published the study result of BTK (Bruton Tyrosine Kinase) inhibitor orelabrutinib in patients with relapsed or refractory (r/r) Marginal Zone Lymphoma (MZL), which investigated the efficacy and safety of orelabrutinib in r/r MZL (Press release, InnoCare Pharma, SEP 12, 2023, View Source [SID1234635125]). The journal concluded that orelabrutinib demonstrated high response rates with durable disease remission and was well tolerated in patients with relapsed or refractory MZL.

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The majority of the enrolled patients had late-stage disease. After a median follow-up duration of 24.3 months, the overall response rate (ORR) assessed by an Independent Review Committee (IRC) ORR was 58.9%. Tumor reduction was observed in 92.2% of patients. The IRC-assessed median duration of response (DOR) was 34.3 months and median progression-free survival (PFS) was not reached. The 12-month PFS rate and overall survival (OS) rate was 82.8% and 91.0% respectively.

Due to the high target selectivity and fewer off-target effects, orelabrutinib demonstrated a good safety profile in the treatment of patients with r/r MZL.

In China, orelabrutinib was the first and only approved BTK inhibitor for the treatment of r/r MZL. Orelabrutinib has been also approved in China for the treatment of r/r chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and r/r mantle cell lymphoma (MCL).

Marginal zone lymphoma (MZL) is a type of non-Hodgkin lymphoma (NHL) of indolent nature originating from B cells in the marginal zones of the spleen, lymphatic tissue, and lymph nodes. MZL accounts for 7%-8% of NHL cases and is the second most prevalent lymphoma among elderly adults. The annual incidence of MZL has increased globally.

The journal concluded that orelabrutinib produced a robust response and was well tolerated in r/r MZL patients. High response rates were consistent among patients with different MZL subtypes and those with negative disease prognostic factors at baseline. The results of this study support the use of orelabrutinib as an effective and tolerable oral treatment option for r/r MZL patients.

The American Journal of Hematology is an academic journal focusing on hematology, which was founded in 1976 and published monthly by WILEY publisher. The journal has been included in SCIE and SCI databases, with an impact factor of 13.268 in 2022.

Note: In addition to background information, the content of this press release is derived from this published article. Full text can be found in View Source

About Orelabrutinib

Orelabrutinib is a highly selective BTK inhibitor developed by InnoCare for the treatment of cancers and autoimmune diseases.

On Dec. 25, 2020, orelabrutinib received conditional approval from the China National Medical Products Administration (NMPA) in two indications: the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL), and the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL). At the end of 2021, orelabrutinib was included into National Reimbursement Drug list to benefit more lymphoma patients. On Nov. 22, 2022, orelabrutinib was approved for the treatment of R/R MCL in Singapore. On April 20, 2023, orelabrutinib was approved for the treatment r/r MZL in China.

In addition to the approved indications, multi-center, multi-indication clinical trials are underway in the US and China with orelabrutinib as monotherapy or in combination therapies, such as first line treatment of MCD subtype of diffuse large B-cell lymphoma (DLBCL).

Orelabrutinib was granted as Breakthrough Therapy Designation for the treatment of r/r MCL by U.S. Food and Drug Administration (FDA). Patient enrollment of Phase II registrational trial for R/R MCL was completed in the U.S. The Company expects to submit the NDA to the U.S. Food and Drug Administration (US FDA) in the middle of 2024.

In addition, the Company has achieved proof of concept (PoC) of orelabrutinib for the treatment of primary immune thrombocytopenia purpura (ITP) and the Phase III registrational trial is ongoing in China. Orelabrutinib’s global phase II studies for the treatment of Multiple Sclerosis (MS), and clinical trials for the treatment of SLE achieved proof of concept (PoC), and orelabrutinib’s phase II study for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD) is ongoing in China.

On September 12, 2023 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, reported that it intends to offer and sell shares of its common stock, and to certain investors, pre-funded warrants to purchase shares of its common stock, in an underwritten public offering (Press release, Rocket Pharmaceuticals, SEP 12, 2023, View Source [SID1234635124]). The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $150 million. The purchase price of each pre-funded warrant will equal the price per share at which shares of common stock are being sold to the public in this offering, minus $0.01, which will be the per share exercise price of each pre-funded warrant. All shares and pre-funded warrants in the offering are to be sold by Rocket. In addition, Rocket intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of shares of its common stock offered in the public offering. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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J.P. Morgan, Morgan Stanley, Leerink Partners and TD Cowen are acting as joint book-running managers, and LifeSci Capital is acting as lead manager for the offering.

The shares and pre-funded warrants are being offered by Rocket pursuant to an effective shelf registration statement that was previously filed with the U.S. Securities and Exchange Commission (the "SEC"). The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at View Source

When available, copies of the final prospectus supplement relating to the offering may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, by telephone at (866) 803-9204, or by email at [email protected]; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, by telephone at (866) 718-1649 or by email at [email protected]; Leerink Partners LLC, Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525 ext. 6105 or by email at [email protected]; or Cowen and Company, LLC, 599 Lexington Avenue, New York, NY 10022, by telephone at (833) 297-2926 or by email at [email protected]. You may also obtain a copy of this document free of charge by visiting the SEC’s website at www.sec.gov.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On September 12, 2023 Mission Bio, a leader in single-cell multi-omic solutions for precision medicine, reported to have launched the Tapestri Genome Editing Solution (Press release, Mission Bio, SEP 12, 2023, View Source [SID1234635123]). Designed to meet the pressing need for high-resolution analysis of genome editing, this first-of-its-kind solution addresses a long-standing gap in advanced therapy development, disease modeling, and functional genomics. Already drawing substantial interest from key opinion leaders in pharmaceuticals, academia, and government labs, this much-anticipated solution was initially previewed at The American Society of Gene and Cell Therapy annual conference.

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The Tapestri Genome Editing Solution has numerous applications for genome engineers, advanced therapy developers, and disease modelers. The solution allows for the rapid characterization of gene-edited drug products, identifying on- and off-target editing, the zygosity of edits, and the co-editing of multiple targets in single cells. The solution also supports multi-omic analysis — co-measuring editing and cell-surface protein expression in the same cells. This paints a clearer picture of cell engineering, as one can determine which cell types are edited, or perhaps validate a knockout through the loss of a protein.

"The Tapestri Genome Editing Solution equips researchers with the capability to scrutinize gene therapies at the single-cell level, a crucial step for ensuring patient safety," said Brittany Enzmann, PhD, Product Manager for Mission Bio’s Tapestri Genome Editing Solution. "By providing this unparalleled resolution, we’re not just offering a new tool; we’re contributing to the evolution of safer and more effective gene therapies. The result is a faster transition from the lab to the patient."

Gene editing technologies, such as CRISPR, have offered immense opportunities for addressing incurable and untreatable diseases like sickle cell disease, where the first CRISPR-edited cell therapy may be approved by the U.S. Food and Drug Administration later this year. Gene editing has also proven to be a powerful strategy in developing disease models and determining genomic drivers of disease progression. However, they also bring intricate challenges such as evaluating the fidelity of both intended and unintended edits. Conventionally, researchers have to perform single-cell cloning in order to fully understand what types of edits were occurring in individual cells, a labor-intensive process that involves cell culture for several weeks.

Mission Bio’s Tapestri Genome Editing Solution was developed to provide a high-throughput workflow that combines genotypic and immunophenotypic assessments in single cells for samples of thousands of cells, capturing crucial details often overlooked by bulk analysis methods. The platform features automated data reporting, eliminating the need for devoted bioinformatics resources and saving days of computational work. As a result, a wider range of professionals, from genome engineers to clinicians to therapy developers, can access and interpret this critical information more readily.

Saar Gill MD, PhD, Associate Professor of Medicine at the University of Pennsylvania, whose laboratory focuses on developing genetically engineered immune cells for the treatment of cancer states, "This multi-omic genome-editing solution will enable critical insights for gene-edited cell therapies beyond what conventional bulk analysis can offer. The automated report gives an immediate and intuitive first look at our data, something that would usually require hours of bioinformatic labor. All in all, we are better equipped to understand our editing results with this technology."

The technology will be showcased at the upcoming Cell Therapy Analytical Development Summit in Amsterdam from September 19-21. As gene editing continues to take on an increasingly critical role in the medical and scientific communities, the spotlight will be on this new technology’s potential to reshape our understanding of cell therapy characterization. Matthew Cato, Vice President, Business and Strategic Market Development at Mission Bio, is scheduled to deliver a presentation aimed at unpacking the technology’s promise and utility.

For more information on the Single-cell Tapestri Genome Editing Solution, visit View Source

On September 12, 2023 Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage oncology company, reported that it has dosed the first patient with MT-302 in a Phase 1 study for advanced or metastatic epithelial tumors (Press release, Myeloid Therapeutics, SEP 12, 2023, View Source [SID1234635122]). MT-302 is the Company’s lead development candidate for in vivo immune cell programming and delivers TROP2-targeting RNA chimeric antigen receptors that express selectively within myeloid cells.

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Dosing with MT-302 represents a significant advance in the development of new therapies for solid tumors, particularly those arising from epithelial tissues. Unlike traditional CAR-T cell therapies, Myeloid’s approach focuses on in vivo programming of immune cells with an off-the-shelf mRNA encoded CAR technology.

TROP2 is a clinically relevant target, as evidenced by clinical findings with several TROP2-targeting antibody-drug conjugates (ADCs). MT-302 advances upon the progress of these existing approaches, by not only targeting TROP2, but also eliciting a comprehensive immune response against the tumor. This immune response is pivotal for sustained immune surveillance and defense against tumor recurrence, aligning with Myeloid’s overarching clinical vision to improve clinical outcomes.

"Initiation of patient dosing with MT-302 is a major milestone for Myeloid in our effort to deliver better treatment options for patients living with solid tumors. We are leading the way with our proprietary approach to in vivo programming, including with many novel CAR constructs designed for selective expression in a wide range of immune cells," said Daniel Getts, Ph.D., CEO of Myeloid. "By advancing MT-302 into the clinic, we are harnessing the power of the innate immune system to overcome many observed limitations of CAR-Ts for solid tumors. We look forward to advancing MT-302 in our Phase 1 study and demonstrating the potential of our innate immunity platform to program cells directly in vivo and drive better outcomes."

Myeloid’s in vivo programming candidates are designed to deliver the highest standard in personalized therapy, providing benefit to patients while reducing time and costs through the elimination of ex-vivo handling of patient cells and complex neoantigen sequencing. The Myeloid platform integrates validated antibody-antigen binding, with novel combinations of myeloid-signaling domains, coded within a simple mRNA that can be delivered repeatedly using LNPs. The platform versatility provides a range of signaling domains and immune cell types useful for combination approaches.

Over 500,000 patients per year in the United States present with TROP2 expressing tumors. Despite the clinical progress observed within the class of TROP2-targeting ADCs, MT-302 goes further, by combining direct tumor targeting and adding immune-stimulation and an adaptive immune response to the tumor neoantigens. The adaptive response is central to long-term immune surveillance and defense against disease recurrence.

About the Phase 1 Study of MT-302

The MT-302 Phase 1 study (NCT05969041) is an open-label dose escalation study to investigate the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of MT-302 in adults with advanced or metastatic epithelial tumors that overexpress TROP2. This study will also define the recommended Phase 2 dose (RP2D) of MT-302. The study is currently enrolling patients at multiple clinical sites in Australia.

About MT-302

MT-302 represents the first candidate in an entirely new, therapeutic modality. It is a first-in-class, TROP2-FcA-LNP, with a strong preclinical profile that supports its advance into this first-in-human trial. TROP2 is overexpressed in most human solid epithelial cancers, with lower expression in corresponding normal tissue. Increased TROP2 expression has been linked to tumor growth.

Treatment with MT-302 demonstrates activity as a monotherapy in a TROP2/TNBC preclinical model, confirming the tumor-fighting potency of programmed myeloid cells even in the absence of T cells. Unlike ADCs, MT-302 brings the potential advantages of eliciting a full immune response by also presenting tumor neoantigen to stimulate T cells. MT-302 has demonstrated strong expression and a favorable safety profile in myeloid cells in rodents and in non-human primates.